• SHCA publishes reports on patient access to treatment

    The SHCA has published two reports analysing the current system in England for determining access to treatment for patients with rare and complex conditions.

    Achieving fairness for all highlights the complexity of appraisal mechanisms for rare disease treatments, involving multiple different methodologies, processes and timelines. For patients and representatives, this complexity creates significant challenges in being able to fully engage with national decision-takers to inform their evaluations. This challenge is compounded by the methodologies used by these national bodies, which do not provide sufficient flexibility to take into account the characteristics common to rare disease treatments of less extensive clinical evidence and relative higher per-patient costs than treatments for more common conditions.

    The SHCA is therefore calling for reform to the system, to address imbalances in the assessment of rare disease treatments. The SHCA has developed a set of principles that should guide national policymakers in delivering this reform:

    • There must be a ‘premium for rarity’ – to enable the system to fairly account for the higher cost of treatments for rare diseases
    • In the event of uncertainty, the presumption should be in favour of access – to enable the system to fairly account for the uncertainty in the evidence base for new treatments, as in the case of the Cancer Drugs Fund
    • The patient voice must be heard – to enable smaller charities to fully engage in processes and inform decision-taking
    • Decisions must be made transparently – to ensure that the system has the confidence of patients
    • The NHS must work with pharmaceutical companies to enable it to take consistent decisions over time – to provide sustainability of funding for treatments for rare diseases

    A Backwards Step? provides an assessment of the April 2017 changes to NICE’s processes and methods, when a ‘Budget Impact Test’ (BIT) for all treatments was created and a cost-effectiveness threshold was introduced to the Highly Specialised Technologies (HST) programme for the first time. The report was developed in response to concerns raised by SHCA members about the changes. In a survey we carried out in April and May this year, 81% of respondents reported that they were either very concerned (47%) or concerned (34%) about the potential impact the reforms could have on patients with rare and complex conditions.

    More than a year on from the changes, the SHCA sought to assess the extent to which these concerns had been realised. Frustratingly, we encountered challenges in carrying out a comprehensive assessment, due to low levels of transparency in the case of the BIT and extended delays in the publication of HST guidance. However, it does appear that the new HST thresholds are impeding patients’ access to treatment.

    The SHCA is therefore calling for the scheduled review of the BIT and HST thresholds to be brought forward and for patients and their representatives to be fully engaged in this process. In the interim period, we believe that the thresholds should be lifted and that NICE should work with NHS England to take steps to improve transparency around the reporting of the application of the BIT.

  • SHCA writes to the three party leaders urging them to commit to reversing NHS medicines rationing

    The Telegraph has published an article on the letter signed by over 30 members of the SHCA on the rationing of treatments. The full text can be found here and has been included below:

    More than 30 charities have written to the three party leaders urging them to commit to reversing NHS measures to increase rationing of medicines.
    Health officials last month brought in new thresholds which mean access to one in five treatments could be delayed or restricted.
    Under the rules, all drugs expected to cost the NHS a total more than £20 million a year will be checked against new “affordability criteria”.
    The cost threshold set by NHS England could affect medicines costing as little eight pence a day, if used commonly enough, as well as high cost medicines used for rare diseases.
    Rationing body the National Institute for Health and Care Excellence (Nice) has said it is likely to affect around 20 per cent of drugs it assesses, with the process including changes that the head of its rationing body has previously described as “unfair”.
    In an open letter to Theresa May, Jeremy Corbyn and Tim Farron, the charities – which include Parkinson’s UK, the Children’s Heart Federation and the MS Society – ask all the parties to commit to reversing the changes.
    The signatories – all members of the Specialised Healthcare Alliance – said that the measures “stand to restrict and ration treatments for people with rare and complex conditions, and were implemented without the agreement of Parliament.”
    The letter comes as political parties prepare to publish their manifestos for the election.
    Caroline Harding, chief executive of Genetic Disorders UK said: “Under these plans, some of the most critically ill patients are being denied access to potentially life-changing treatments.
    “Any decision to ration access to medicines should be taken by ministers, not unelected officials.”
    Kay Boycott, chief executive, Asthma UK, said: “There is a real risk that these plans will turn the clock back on access to life transforming treatment.”
    Sarah Vibert, chief executive, Neurological Alliance, said: “Politicians from all sides should pledge to rethink these damaging proposals.
    “It is completely unacceptable that patients with neurological conditions risk being denied access to treatment solely on the basis of cost.
    None of the parties have formally published their manifestos. But the draft Labour manifesto, leaked last week, said: “Labour will tackle the growing problem of rationing of services and medicines across England, taking action to address postcode lotteries and making sure that the quality of care you receive does not depend on which part of the country you live in. We will ensure that NHS patients get fast access to the most effective new drugs and treatments.”
    Jonathan Ashworth, shadow Health Secretary, said: “There is a real and growing worry that Theresa May’s ongoing underfunding of the NHS means patients just aren’t getting access to new medicines. Patients need fast access to medicines and treatments which are recommended by Nice as being clinically and cost effective. Labour is committed removing the barriers which are being put in place and to ensuring that NHS patients get fast access to the most effective new drugs and treatments.”
    A Conservative spokesman said: “NHS spending on medicines and treatments is now second only to staffing costs – in fact, the NHS in England spent more than £15 billion on medicines last year, a rise of nearly 20 per cent since Labour left office. But we can only ensure more patients than ever get outstanding care if we continue to invest in the NHS on the back of a strong economy – something Jeremy Corbyn and the other parties who’d prop him up in Downing Street simply could not do.”
    Liberal Democrat Health Spokesperson Norman Lamb said:  “This is a shameful retreat from the core principle of the NHS, that treatment should be available to patients regardless of their ability to pay.
    “The rationing of treatments is an inevitable consequences of this government’s chronic underfunding of the NHS. NHS England is caught between a rock and a hard place because they simply don’t have enough resources.

  • SHCA raises concerns about increasing medicines rationing

    In a letter to the Editors of The Times, members of the SHCA raised their deep concerns with the reforms to NICE and called for all politicians standing for 2017 General Election to commit in their manifestos to reverse these recent reforms.

    The full letter can be found here and has been included below:

    Sir, We want to see an NHS that provides high-quality care, support and treatment to everyone who needs it — and to ensure that our voice is heard during the general election campaign. In particular, we want all politicians standing for election to know of our deep concern with the reforms to the National Institute for Health and Care Excellence (Nice) that the government and NHS England implemented from April 1. These reforms stand to restrict and ration treatments for people with rare and complex conditions, and were implemented without the agreement of parliament.

    With that in mind, we urge political parties to commit in their manifestos to reverse these recent reforms, and to guarantee that any future reforms will be considered by parliament before being implemented. We also ask that any decisions to restrict the availability of Nice-approved treatments are taken by democratically-elected politicians.
    Deborah Bent, Charity Manager, Limbless Association; David Bickers, CEO, Douglas Bader Foundation; Kay Boycott, CEO, Asthma UK; Roger Brown, Chair, Waldenstrom’s Macroglobulinemia UK; Nic Bungay, Director of Campaigns, Care and Information, Muscular Dystrophy UK; Liz Carroll, CEO, The Haemophilia Society; Tanya Collin-Histed, CEO, Gauchers Association; Ann Chivers, CEO, Alström Syndrome UK; Genevieve Edwards, Director of External Affairs, MS Society; Sue Farrington, CEO, Scleroderma & Raynaud’s UK; Steve Ford, CEO, Parkinson’s UK; Kye Gbangbola, Chair, Sickle Cell Society; Deborah Gold, CEO, National AIDS Trust; Caroline Harding, CEO, Genetic Disorders UK; Tess Harris, CEO, The Polycystic Kidney Disease Charity; Dr Lesley Kavi, Postural Tachycardia Syndrome UK (PoTS UK); Anne Keatley-Clarke, CEO, Children’s Heart Federation; Caroline Morrice, CEO, GAIN; Allan Muir, Development Director and Type II Co-ordinator, Association for Glycogen Storage Disease (UK); Patricia Osborne, CEO, Brittle Bone Society; Jill Prawer, Founder and Chair, LPLD Alliance; Lynne Regent, CEO, Anaphylaxis Campaign; Richard Rogerson, Niemann-Pick UK; David Ryner, The CML Support Group; Timothy Statham OBE, CEO, National Kidney Federation; Laura Szutowicz, CEO, HAE UK; Paddy Tabor, CEO, British Kidney Patient Association; Jeremy Taylor, CEO, National Voices; Oliver Timmis, CEO, Alkaptonuria (AKU) Society; Gabriel Theophanous, President, UK Thalassaemia Society; Sarah Vibert, CEO, The Neurological Alliance; Dr Susan Walsh, Director, Primary Immunodeficiency UK

  • SHCA sends letter to the Prime Minister on restricting and rationing treatment

    30 members of the SHCA have sent a letter to the Prime Minister raising the alarm about restrictions and rationing of treatment because of underfunding, especially for patients with rare and complex conditions

    The letter was covered in the Observer newspaper. The article can be found here and is included below:

    Charities call for NHS to stop rationing critical care

    Theresa May has been urged by charity chiefs to stop the NHS rationing treatment for seriously ill patients and to find more money for their care in the budget next month.

    About 30 health charities, including the Teenage Cancer Trust, National AIDS Trust and Motor Neurone Disease Association, have raised the alarm about NHS England “restricting and rationing treatment” because of underfunding, especially for patients with rare and complex conditions. The groups from the Specialised Healthcare Alliance said this rationing is taking place without sufficient public scrutiny.

    In a letter to the prime minister, they said NHS England is “choosing to restrict and ration treatments that patients with rare and complex conditions need – often without proper consultation with patients”. “We hope that you will take action to ensure full patient involvement in these decisions, and to ensure that any decision to ration treatment is overseen by democratically elected politicians,” they added.

    The warning comes after health thinktank the King’s Fund said that politicians would be escaping culpability for rationing decisions, as plans due to come into force from April mean that even cost-effective treatments could be denied to patients.

    Nicholas Timmins, a policy adviser at the organisation, wrote: “If the NHS can no longer fund new and cost-effective treatments, ministers should announce that decision case by case, and be held accountable for so doing”.

    It emerged last month that cancer, diabetes and asthma patients could all be hit by the new affordability criteria, which means that drugs which cost the NHS more than £20m in total each year could be subject to restrictions.

    The charity chiefs also raised wider concerns about funding for the NHS, which medics have said is under huge pressure this winter because of cuts to adult social care provided by councils.

    Philip Hammond, the chancellor, is facing calls to give more money to the NHS next month after resisting an emergency bailout at the autumn statement. Instead, the government has launched a long-term review of social care and will allow councils to raise more through a local ‘precept’ (an extra charge to council tax bills which can only be used for adult social services) in the next couple of years.

    No 10 and the Treasury appear to remain firmly against repeated calls from medics and the opposition for more cash for the NHS, indicating they are not prepared to change tack at the budget. But the charities say: “Our charities – and the patients we represent – are deeply concerned by the recent statements of Simon Stevens concerning the NHS’s relative underfunding. We urge you to ensure that this year’s budget provides the NHS with the funding it needs to deliver the standards of care patients expect.”

    The charities issued their warning ahead of a Lords debate on the medical supplies bill this week, which Labour’s shadow health minister Lord Philip Hunt has described as a missed opportunity to “get rid of many of the restrictions on NHS patients using innovative new medicines”. A government spokesman said blanket restrictions on treatment are unacceptable and that the clinical needs of a patient, and the urgency with which they require treatment, must come first.

    “The government is responding to the needs of the NHS by investing more money – £4bn this year – to fund its own plans for the future”.

  • SHCA raises concerns about NHS England's generic commissioning policy proposals

    NHS England is currently consulting on proposed changes to a suite of generic commissioning policies. These include the provisions for dealing with individual patient funding requests, and how national commissioning policies are developed outside the annual commissioning cycle.

    The Specialised Healthcare Alliance has campaigned for the review of these policies for several years and, while welcoming the belated consultation, is concerned at the proposed approach. This risks seeing impractically high barriers established for individual patient funding applications, and omits consideration of clinically critically urgent cases.

    The Alliance’s consultation response can be read in full here: SHCA response to NHS England generic policies consultation

  • SHCA responds to NICE and NHS England consultation on changes to technology appraisals

    The Specialised Healthcare Alliance has responded to NICE and NHS England’s joint consultation on significant changes to health technology appraisals and highly specialised technology appraisals.

    Overall, the Alliance is concerned at the consultation proposals. In particular, the provisions for highly specialised technologies fall significantly short of what is required and risk jeopardising access to innovation for very small patient populations in future.

    The SHCA’s full response can be read here: SHCA response to NICE and NHS England consultation on changes to technology appraisals and highly specialised technology appraisals

  • SHCA signs #PatientsTogether letter to The Times

    The Specialised Healthcare Alliance has joined other patient representative organisations in signing a letter to the editor of The Times newspaper calling for NHS England to communicate its prioritisation decisions respectfully to all.

    The letter is as follows.

    “Sir, Over recent weeks, we have been alarmed by the “divide and rule” tactics used by NHS England when communicating which new treatments it will make available to patients.

    These funding decisions affect the lives of people with a range of rare or complex conditions. As organisations representing the patients who would benefit from these treatments, we are concerned by NHS England pitting the interests of one patient group against another in its press statements. All patients and all conditions matter.

    In the light of this week’s High Court hearing about the HIV prevention drug, PrEP, NHS England will soon look again at which treatments it will fund.

    We understand the NHS must make difficult choices. All we ask from our national health service is to adopt an ethical and transparent approach in taking these decisions and to communicate them in a way that is respectful to the people who are affected by them.

    Jeremy Taylor, chief executive, National Voices
    John Murray, director, Specialised Healthcare Alliance
    Ian Green, chief executive, Terrence Higgins Trust
    Steve Ford, chief executive, Parkinson’s UK
    Sally Light, chief executive, Motor Neurone Disease Association
    Philip Lee, chief executive, Epilepsy Action
    Charles Gore, chief executive, Hepatitis C Trust
    Menai Owen-Jones, chief executive, The Pituitary Foundation
    Roger Brown, chair, Waldenstrom Macroglobulinemia UK”

  • SHCA welcomes Public Accounts Committee recommendations for NHS specialised services

    The SHCA today welcomes the publication of the House of Commons Public Accounts Committee’s report on NHS Specialised Services, following a recent evidence session and National Audit Office report on the issue.

    To a significant extent, the report reflects the views of the Alliance and its members, as expressed through submissions to the inquiry. The Committee sets out areas of significant concern, including NHS England’s lack of transparency and accountability, poor data and business intelligence, and need for stronger financial control.

    The Committee’s recommendations include:

    1. As a matter of urgency, NHS England should ensure that a consistent process is put in place to ensure its decision-making is transparent and equitable. It must improve the transparency of its decision-making by publishing a document, by September 2016, which sets out the roles of its advisory committees and decision-making bodies, the decisions they make, how these decisions will be documented, and when and to whom they will be made available.
    2. NHS England should engage with clinical commissioning groups to address barriers to collaborative commissioning and, by October 2016, set clear milestones and timelines by which measurable service change and patient benefit from this initiative will be demonstrated.
    3. NHS England has said it will be collecting more consistent data. By April 2017, it should use this data to link spend, by service provided, to service quality, patient outcomes and patient experience; to allow clear comparison between different providers and to improve value for money.

    While some of the Committee’s concerns on the large increases in budget for specialised commissioning since April 2013 can be explained by the incorrect baseline budget set at the time of NHS England’s establishment, a range of concerns are set out in relation to future cost growth and management of provider activity.

    The Alliance will press for NHS England to deliver on the Committee’s recommendations in the months ahead and looks forward to ensuring that patients’ interests are at the front and centre of this work. The Alliance considers that the interrelated recommendations of the Specialised Services Commission published in May provide a practical template for NHS England.

  • HSJ article on the changes underway in specialised services

    The Health Service Journal has published a comment piece by the Deputy Director of the Specialised Healthcare Alliance on the strategic changes underway in specialsied commissioning.

    The original article can be read on the HSJ website, here. The text is also available here

  • Lord Warner's Specialised Services Commission Publishes Final Report

    An expert commission on specialised services, drawn from across the health service and chaired by Lord Warner, has called for major changes to improve outcomes and efficiency but warned that these alone are unlikely to avoid hard choices about what the NHS can afford without improved funding.

    Specialised services costing £15 billion a year encompass everything from severe burns and spinal injury to treatments for cancer and a growing number of rare diseases. As such they are fundamental to a comprehensive health service and essential to keeping British clinical practice in the vanguard of global medicines.

    In the wake of a recent NAO report, the commission strongly supports the need for national funding and accountability for these strategic services and sees the associated mandatory national standards as a key means of liberating more local approaches to management and delivery.

    In particular, the commission recommends greater freedom for commissioners to work with networks of providers to deliver end to end services for patients’ entire care, enabling them to minimise use of expensive and overstretched hospital resources. New models of remuneration should reflect payment by outcomes for the patient rather than hospital-based activity, underpinned by sound data.

    The commission considers that specialised services should play their part in maximising the value of every pound spent by the NHS but doubts that efficiency improvements will be enough to avoid pushing the NHS towards unwanted rationing of services, treatments or both.

    Lord Warner comments: “The NHS is a complex eco-system running from general practice through to world-leading centres of expertise and excellence. The commission favours more efficient joint-working through networks, making patients rather than hospitals the hub of care. The current danger is that chronic deficits will progressively impede the range and quality of what the NHS can afford to do. This would have ramifications for patients and this important part of the UK economy alike.”


    1. Lord Warner served as a Health Minister in the Blair government from 2003 to 2007 and advised the Coalition Government from 2010 to 2015 in various capacities. He now sits as a non-affiliated peer.
    2. The Specialised Services Commission was convened by Lord Warner on the 10th anniversary of the publication of the Carter Review of Specialised Commissioning. The full membership was:

    Professor Dame Sue Bailey (Chair, Academy of Medical Royal Colleges)
    Professor Maureen Baker (Chair, Royal College of General Practitioners)
    Robin Bhattacherjee (General Manager, Actelion – representing the ABPI)
    Professor Tim Briggs (National Director of Clinical Quality and Efficiency, Department of Health)
    Chris Hopson (Chief Executive, NHS Providers)
    Gina Lawrence (Chief Operating Officer, NHS Trafford CCG)
    Professor Jane Maher (Joint Chief Medical Officer, Macmillan Cancer Support)
    Ed Owen (Chief Executive, Cystic Fibrosis Trust)
    Richard Rogerson (Trustee, Niemann Pick UK)
    Julie Wood (Chief Executive, NHS Clinical Commissioners)


    Deirdre Evans (Director, Scottish National Services Division)
    Fiona Marley (Head of Highly Specialised, NHS England)
    Daniel Phillips (Director of Specialised Services, NHS Wales)

    3. The Commission conducted its work between December 2015 to April 2016 and was assisted by written evidence from a variety of patient organisations, Royal Colleges, industry bodies and companies.
    4. The Commission’s recommendations are attached as an Appendix.
    5. The National Audit Office published its report into NHS England’s delivery of specialised services commissioning on 27th April 2016. The report can be found here.
    6. The Commission has been serviced by the Specialised Healthcare Alliance’s secretariat. JMC Partners currently provides the secretariat to the Specialised Healthcare Alliance.
    7. For further information on the Commission’s report, please contact Mark Loughridge (

    Specialised Services Commission – Final Report